| Date & Time | 1-5 June 2026 |
|---|---|
| Location | Utrecht, The Netherlands |
A 5-day course offering an end-to-end translational overview, from discovery through manufacturing and regulation, to reimbursement and patient access
HOW DOES THE COURSE WORK?
This interactive 5-day course is focused on how ATMPs can be successfully translated from the laboratory to the clinic, looking specifically into Scientific principles, Manufacturing/CMC, Regulatory pathways, Reimbursement and Patient access. You will learn from real-life scenarios presented by experts in the field, and group assignments. Before attending the course, an 8-hour e-learning course will equip you with the essential knowledge to make the most of the course.
WHO IS THE COURSE FOR?
The course is designed for rare-disease professionals, including researchers, clinicians, project/programme managers and other professionals involved in the ATMP development process. It is suitable for individuals at different career stages from late-stage PhD students to Principal Investigators and in different settings (academia, small and medium enterprises, industry, philanthropy).
To support the adaptation of this course to new target groups or geographic areas, we will welcome interested parties to join the course as observer.
What will I learn?
Focusing on Scientific principles, Manufacturing/CMC, Regulatory pathways, Reimbursement and Patient access, the course will meet the following learning objectives, where participants attending the course will be able to:
- Provide an overview of the ATMP development cycle and define the major bottlenecks within the cycle
- Know where to access further knowledge / information to support when developing ATMPs for Rare Disease
What are the costs?
The course is free to attend thanks to ERDERA funding and includes daytime catering and a dinner.
Please note that travel and accommodation are not included but ERDERA will provide two travel fellowships for participants from EU-13 countries and ERDERA underrepresented countries.*
*Participants based in Bulgaria (BG), Croatia (HR), Cyprus (CY), Czechia (CZ), Estonia (EE), Greece (GR), Georgia (GE), Hungary (HU), Ireland (IE), Latvia (LV), Lithuania (LT), Malta (MT), Morocco (MA), Poland (PL), Portugal (PT), Romania (RO), Serbia (RS), Slovakia (SK), Slovenia (SI) and Türkiye *(TR).
How to apply?
To apply for the course, you will be asked to provide some details, including a brief outline of your current research, your motivation for attending the course, and how your participation might benefit you and the Rare Disease community. We are only accepting 30 participants, who will be selected by the curriculum committee. As part of the registration process, you can also apply for a travel fellowship. Registration closed on 28.02.2026.
About the course organisers
EATRIS is a non-profit organisation bringing together people, resources and services to translate scientific discoveries into benefits for patients. ERDERA – the European Rare Diseases Research Alliance – is a European partnership uniting over 170 public and private organisations across 37 countries around a single goal: turning cutting-edge science into tangible benefits for the thirty million Europeans living with a rare disease funded by the European Union’s Horizon Europe research and innovation programme under grant agreement N°101156595.
Course programme (subject to change)
| 1 June | Scientific principles and translational path |
| 12:00-17:00 | Genetropolis board game – Develop an ATMP from idea to patient access and learn about pitfalls preventing successful translation |
| Scientific principles of Gene editing, (next generation) CAR-T cells and non-viral approaches | |
| 19:00 | Course dinner |
| 2 June | ATMP manufacturing |
| 09:00-17:00 | Good manufacturing practice |
| Quality assurance in ATMP manufacturing | |
| AI to support ATMP manufacturing | |
| Gene Delivery | |
| 3 June | Regulatory pathways |
| 09:00-17:00 | Introduction to EMA regulations |
| Beyond Traditional Clinical Trials | |
| Regulatory sandboxes & masterfiles | |
| 4 June | Reimbursement & patient access |
| 09:00-17:00 | Health Technology assessment |
| Biosimilars for ATMPs | |
| Patient engagement in research | |
| 5 June | Platform technologies for rare diseases |
| 09:00-15:00 | Challenges and support when developing ATMPs for rare diseases |
| Stakeholder engagement and career networks | |
| IP in an academic context |
Faculty
- Ana Hidalgo-Simon, Scientist and regulator, Leiden University Medical Center
- David Morrow, Scientific lead for Advanced Therapies, EATRIS
- Elena Guillen Benitez, Medical Doctor, Clinical Pharmocology specialist, Leiden University Medical Center
- Harry Dolstra, Radboud University Medical Center
- Maria Themeli, Assistant professor, Co-chair EATRIS ATMP platform, Amsterdam University Medical Center
- Renske ten Ham, Assistant Professor, Health Economics & HTA of Regenerative Medicines, Utrecht University Medical Center
- Rosan Vegter, Scientific lead Rare Diseases and Regulatory Advisory, EATRIS
- Trudy Straetemans, Associate Professor Hematology department and PI Center for Translational Immunology, University Utrecht
